325. 遺伝性自己炎症疾患
［臨床試験数：6，薬物数：10（DrugBank：6），標的遺伝子数：2，標的パスウェイ数：33］
Searched query = "Hereditary autoinflammatory syndrome", "NLRC4 abnormality", "Adenosine deaminase 2 deficiency", "ADA2 deficiency", "Aicardi-Goutieres syndrome", "A20 haploinsufficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03304717 (ClinicalTrials.gov) | January 2021 | 4/8/2017 | Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome | Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome | Aicardi Goutières Syndrome | Drug: Tenofovir (TDF) and Emtricitabine (FTC);Other: Placebo | Children's Hospital of Philadelphia | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);National Human Genome Research Institute (NHGRI);Gilead Sciences;Emerson Resources;National Institutes of Health (NIH) | Not yet recruiting | 2 Years | 18 Years | All | 34 | Phase 1;Phase 2 | United States |